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CRISPR/Cas9系统:递送技术与生物医学应用

CRISPR/Cas9 systems: Delivery technologies and biomedical applications.

作者信息

Du Yimin, Liu Yanfei, Hu Jiaxin, Peng Xingxing, Liu Zhenbao

机构信息

Department of Pharmaceutics, Xiangya School of Pharmaceutical Sciences, Central South University, Changsha 410013, China.

Department of Pharmaceutical Engineering, College of Chemistry and Chemical Engineering, Central South University, Changsha 410083, China.

出版信息

Asian J Pharm Sci. 2023 Nov;18(6):100854. doi: 10.1016/j.ajps.2023.100854. Epub 2023 Oct 21.

Abstract

The emergence of the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) genome-editing system has brought about a significant revolution in the realm of managing human diseases, establishing animal models, and so on. To fully harness the potential of this potent gene-editing tool, ensuring efficient and secure delivery to the target site is paramount. Consequently, developing effective delivery methods for the CRISPR/Cas9 system has become a critical area of research. In this review, we present a comprehensive outline of delivery strategies and discuss their biomedical applications in the CRISPR/Cas9 system. We also provide an in-depth analysis of physical, viral vector, and non-viral vector delivery strategies, including plasmid-, mRNA- and protein-based approach. In addition, we illustrate the biomedical applications of the CRISPR/Cas9 system. This review highlights the key factors affecting the delivery process and the current challenges facing the CRISPR/Cas9 system, while also delineating future directions and prospects that could inspire innovative delivery strategies. This review aims to provide new insights and ideas for advancing CRISPR/Cas9-based delivery strategies and to facilitate breakthroughs in biomedical research and therapeutic applications.

摘要

成簇规律间隔短回文重复序列(CRISPR)/CRISPR相关蛋白9(Cas9)基因组编辑系统的出现,给人类疾病治疗、动物模型建立等领域带来了重大变革。为了充分发挥这一强大基因编辑工具的潜力,确保其高效、安全地递送至靶位点至关重要。因此,开发针对CRISPR/Cas9系统的有效递送方法已成为一个关键研究领域。在这篇综述中,我们全面概述了递送策略,并讨论了它们在CRISPR/Cas9系统中的生物医学应用。我们还对物理、病毒载体和非病毒载体递送策略进行了深入分析,包括基于质粒、信使核糖核酸和蛋白质的方法。此外,我们阐述了CRISPR/Cas9系统的生物医学应用。本综述强调了影响递送过程的关键因素以及CRISPR/Cas9系统当前面临的挑战,同时还描绘了可能激发创新递送策略的未来方向和前景。本综述旨在为推进基于CRISPR/Cas9的递送策略提供新的见解和思路,并促进生物医学研究和治疗应用取得突破。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7df6/10711398/c3337952556a/ga1.jpg

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